Literature summary for 3.2.1.76 extracted from

Giugliani, R.; Rojas, V.M.; Martins, A.M.; Valadares, E.R.; Clarke, J.T.; Goes, J.E.; Kakkis, E.D.; Worden, M.A.; Sidman, M.; Cox, G.F.
A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I (2009), Mol. Genet. Metab., 96, 13-19.

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Application

Application	Comment	Organism
medicine	currently approved laronidase dose regimen, to treat the lysosomal storage disorder mucopolysaccharidosis type I, has similar efficacy and potentially improved safety compared to regimens using higher doses, regardless of dose frequency. The approved 0.58 mg/kg/week laronidase dose regimen provides near-maximal reductions in glycosaminoglycan storage and the best benefit-to-risk ratio. The 1.2 mg/kg every 2 weeks regimen may be an acceptable alternative for patients with difficulty receiving weekly infusions, but the long-term effects of this regimen are unknown. In general, laronidase therapy is safe and well tolerated in all treatment groups	Homo sapiens

Localization

Localization	Comment	Organism	GeneOntology No.	Textmining

Organism

Organism	UniProt	Comment	Textmining
Homo sapiens	-	-	-

Synonyms

Synonyms	Comment	Organism
Aldurazyme	-	Homo sapiens
alpha-L-iduronidase	-	Homo sapiens
laronidase	-	Homo sapiens

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Release 2023.1 (January 2023)